Ciltacabtagene autoleucel Medicine 08 December 2020 Extended indication Adult patients with relapsed or refractory multiple myeloma, whose prior regimens included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody and who had disease progression on the last regimen. Therapeutic value No estimate possible yet Total cost € 16,250,000.00 Registration phase Registration application pending Product Active substance Ciltacabtagene autoleucel Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Multiple Myeloma Extended indication Adult patients with relapsed or refractory multiple myeloma, whose prior regimens included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody and who had disease progression on the last regimen. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Janssen Portfolio holder Mechanism of action CAR-T therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Autologe humane T cellen die ex-vivo genetisch gemodificeerd zijn met een lentivirale vector die codeert voor een chimeric antigen receptor (CAR) tegen het B-cell maturation antigen (BCMA). Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date May 2021 Expected Registration January 2022 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Fabrikant verwacht indiening in de eerste helft van 2021 en registratie in 2022. Therapeutic value Current treatment options Chemotherapie combinaties en in de toekomst mogelijk Idecabtagene vicleucel. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er is nog onvoldoende data beschikbaar om de therapeutische waarde van JNJ-4528 in vergelijking met idecabtagene vicleucel en chemotherapeutische regimes te kunnen bepalen. Hiervoor zullen eerst de langere termijn data van de CARTITIUDE en de KarMMa studie moeten volgen. De eerste data van de CARTITUDE fase 1b studie laten een ORR van 100% zien met een 86% van de patiënten met een stringente CR. De PFS bij 9 maanden was 86%. Echter de mediane follow up is slechts 11,5 maanden. Duration of treatment Frequency of administration Dosage per administration References phase Ib/II CARTITUDE-1 trial (NCT03548207). Additional remarks Expected patient volume per year Patient volume < 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References expert opinie Additional remarks Gezien de vele behandelopties komen naar schatting maximaal 50 patiënten per jaar hiervoor in aanmerking. Naar de toekomst toe kan CAR-T mogelijk naar eerdere behandelrichtlijnen verschuiven, in dat geval zouden meer patiënten in aanmerking komen. Expected cost per patient per year Cost € 320,000.00 - 330,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References https://icer-review.org/announcements/icer-to-assess-car-t-cell-therapies-for-multiple-myeloma/ Additional remarks Nog geen prijs bekend. Wel volgt er volgend jaar een ICER rapport over de waarde van dit product. Huidige inschatting op basis van andere CAR-T behandelingen tisagenlecleucel en axicabtagene ciloleucel (€320.000-€330.000). Potential total cost per year Total cost € 16,250,000.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References adisinsight Additional remarks Indication extension Indication extensions No Indication extensions References AdisInsight Additional remarks Other information Additional remarks